Thirty-six million Europeans — including more than one million in the Nordics[1] — live with a rare disease.[2] For patients and their families, this is not just a medical challenge; it is a human rights issue. Diagnostic delays mean years of worsening health and needless suffering. Where treatments exist, access is far from guaranteed. Meanwhile, breakthroughs in genomics, AI and targeted therapies are transforming what is possible in health care. But without streamlined systems, innovations risk piling up at the gates of regulators, leaving patients waiting. Even the Nordics, which have some of the strongest health systems in the world, struggle to provide fair and consistent access for rare-disease patients. Expectations should be higher. THE BURDEN OF DELAY The toll of rare diseases is profound. People living with them report health-related quality-of-life scores 32 percent lower than those without. Economically, the annual cost per patient in Europe — including caregivers — is around €121,900.[3] > Across Europe, the average time for diagnosis is six to eight years, and > patients continue to face long waits and uneven access to medications. In Sweden, the figure is slightly lower at €118,000, but this is still six times higher than for patients without a rare disease. Most of this burden (65 percent) is direct medical costs, although non-medical expenses and lost productivity also weigh heavily. Caregivers, for instance, lose almost 10 times more work hours than peers supporting patients without a rare disease.[4] This burden can be reduced. European patients with access to an approved medicine face average annual costs of €107,000.[5] Yet delays remain the norm. Across Europe, the average time for diagnosis is six to eight years, and patients continue to face long waits and uneven access to medications. With health innovation accelerating, each new therapy risks compounding inequity unless access pathways are modernized. PROGRESS AND REMAINING BARRIERS Patients today have a better chance than ever of receiving a diagnosis — and in some cases, life-changing therapies. The Nordics in particular are leaders in integrated research and clinical models, building world-class diagnostics and centers of excellence. > Without reform, patients risk being left behind. But advances are not reaching everyone who needs them. Systemic barriers persist: * Disparities across Europe: Less than 10 percent of rare-disease patients have access to an approved treatment.[6] According to the Patients W.A.I.T. Indicator (2025), there are stark differences in access to new orphan medicines (or drugs that target rare diseases).[7] Of the 66 orphan medicines approved between 2020 and 2023, the average number available across Europe was 28. Among the Nordics, only Denmark exceeded this with 34. * Fragmented decision-making: Lengthy health technology assessments, regional variation and shifting political priorities often delay or restrict access. Across Europe, patients wait a median of 531 days from marketing authorization to actual availability. For many orphan drugs, the wait is even longer. In some countries, such as Norway and Poland, reimbursement decisions take more than two years, leaving patients without treatment while the burden of disease grows.[8] * Funding gaps: Despite more therapies on the market and greater technology to develop them, orphan medicines account for just 6.6 percent of pharmaceutical budgets and 1.2 percent of health budgets in Europe. Nordic countries — Sweden, Norway and Finland — spend a smaller share than peers such as France or Belgium. This reflects policy choices, not financial capacity.[9] If Europe struggles with access today, it risks being overwhelmed tomorrow. Rare-disease patients — already facing some of the longest delays — cannot afford for systems to fall farther behind. EASING THE BOTTLENECKS Policymakers, clinicians and patient advocates across the Nordics agree: the science is moving faster than the systems built to deliver it. Without reform, patients risk being left behind just as innovation is finally catching up to their needs. So what’s required? * Governance and reforms: Across the Nordics, rare-disease policy remains fragmented and time-limited. National strategies often expire before implementation, and responsibilities are divided among ministries, agencies and regional authorities. Experts stress that governments must move beyond pilot projects to create permanent frameworks — with ring-fenced funding, transparent accountability and clear leadership within ministries of health — to ensure sustained progress. * Patient organizations: Patient groups remain a driving force behind awareness, diagnosis and access, yet most operate on short-term or volunteer-based funding. Advocates argue that stable, structural support — including inclusion in formal policy processes and predictable financing — is critical to ensure patient perspectives shape decision-making on access, research and care pathways. * Health care pathways: Ann Nordgren, chair of the Rare Disease Fund and professor at Karolinska Institutet, notes that although Sweden has built a strong foundation — including Centers for Rare Diseases, Advanced Therapy (ATMP) and Precision Medicine Centers, and membership in all European Reference Networks — front-line capacity remains underfunded. “Government and hospital managements are not providing  resources to enable health care professionals to work hands-on with diagnostics, care and education,” she explains. “This is a big problem.” She adds that comprehensive rare-disease centers, where paid patient representatives collaborate directly with clinicians and researchers, would help bridge the gap between care and lived experience. * Research and diagnostics: Nordgren also points to the need for better long-term investment in genomic medicine and data infrastructure. Sweden is a leader in diagnostics through Genomic Medicine Sweden and SciLifeLab, but funding for advanced genomic testing, especially for adults, remains limited. “Many rare diseases still lack sufficient funding for basic and translational research,” she says, leading to delays in identifying genetic causes and developing targeted therapies. She argues for a national health care data platform integrating electronic records, omics (biological) data and patient-reported outcomes — built with semantic standards such as openEHR and SNOMED CT — to enable secure sharing, AI-driven discovery and patient access to their own data DELIVERING BREAKTHROUGHS Breakthroughs are coming. The question is whether Europe will be ready to deliver them equitably and at speed, or whether patients will continue to wait while therapies sit on the shelf. There is reason for optimism. The Nordic region has the talent, infrastructure and tradition of fairness to set the European benchmark on rare-disease care. But leadership requires urgency, and collaboration across the EU will be essential to ensure solutions are shared and implemented across borders. The need for action is clear: * Establish long-term governance and funding for rare-disease infrastructure. * Provide stable, structural support for patient organizations. * Create clearer, better-coordinated care pathways. * Invest more in research, diagnostics and equitable access to innovative treatments. Early access is not only fair — it is cost-saving. Patients treated earlier incur lower indirect and non-medical costs over time.[10] Inaction, by contrast, compounds the burden for patients, families and health systems alike. Science will forge ahead. The task now is to sustain momentum and reform systems so that no rare-disease patient in the Nordics, or anywhere in Europe, is left waiting. -------------------------------------------------------------------------------- [1] https://nordicrarediseasesummit.org/wp-content/uploads/2025/02/25.02-Nordic-Roadmap-for-Rare-Diseases.pdf [2] https://nordicrarediseasesummit.org/wp-content/uploads/2025/02/25.02-Nordic-Roadmap-for-Rare-Diseases.pdf [3] https://media.crai.com/wp-content/uploads/2024/10/28114611/CRA-Alexion-Quantifying-the-Burden-of-RD-in-Europe-Full-report-October2024.pdf [4] https://media.crai.com/wp-content/uploads/2024/10/28114611/CRA-Alexion-Quantifying-the-Burden-of-RD-in-Europe-Full-report-October2024.pdf [5] https://media.crai.com/wp-content/uploads/2024/10/28114611/CRA-Alexion-Quantifying-the-Burden-of-RD-in-Europe-Full-report-October2024.pdf [6] https://www.theparliamentmagazine.eu/partner/article/a-competitive-and-innovationled-europe-starts-with-rare-diseases? [7] https://www.iqvia.com/-/media/iqvia/pdfs/library/publications/efpia-patients-wait-indicator-2024.pdf [8] https://www.iqvia.com/-/media/iqvia/pdfs/library/publications/efpia-patients-wait-indicator-2024.pdf [9] https://copenhageneconomics.com/wp-content/uploads/2025/09/Copenhagen-Economics_Spending-on-OMPs-across-Europe.pdf [10] https://media.crai.com/wp-content/uploads/2024/10/28114611/CRA-Alexion-Quantifying-the-Burden-of-RD-in-Europe-Full-report-October2024.pdf Disclaimer POLITICAL ADVERTISEMENT * The sponsor is Alexion Pharmaceuticals * The entity ultimately controlling the sponsor: AstraZeneca plc * The political advertisement is linked to policy advocacy around rare disease governance, funding, and equitable access to diagnosis and treatment across Europe More information here.

“I would like to be able to escape. I dream of an elderly life where I wouldn’t need crutches, and the disappearance of thoughts about having to. I would really, really like to not worry about that,” says Martin Nielsen, who lives in Denmark and has hemophilia B.  Thursday Apr. 17, 2025, is World Hemophilia Day, established to recognize and raise awareness about hemophilia and other bleeding disorders. There are two types of hemophilia — A and B — which are both rare diseases caused by mutations in the genes that control blood clotting. Hemophilia affects around 350,000 women and 1.1 million men worldwide.1,2  Living with hemophilia means navigating life under the threat of spontaneous bleeds — in joints, muscles or, in rare circumstances, the brain — from day-to-day activities or pursuit of a full life.3 As such, people with hemophilia can be confronted with regular thoughts like, ‘what is my risk today?’, along with fears, frustrations and the burden of painful and invasive treatments that can have a mental and emotional toll.3,4   Positively, scientific advances in hemophilia care have opened opportunities for patients to better manage the physical manifestations of their disease.5 Now, the community is focused on reducing the psychological burden, so that patients can achieve a ‘hemophilia-free mind’.6 Crucially, this requires collaboration between patients and their healthcare professionals, encompassing not just physical symptoms, but also the patient’s psychological wellbeing, goals and preferences.  Here, Martin tells his story of living with hemophilia B. Alongside him, Wolfgang Miesbach, professor of medicine at Frankfurt University Hospital, Germany, and member of the executive committee at the European Association for Haemophilia and Allied Disorders, offers insights into the treatment decisions he makes with his patients. This is Martin’s personal story — his experience may not be representative of all people living with hemophilia. Martin Nielsen (left) and Professor Wolfgang Miesbach (right) | via CSL Behring Navigating uncertainty: The rhythm of life with hemophilia  Martin was diagnosed when he was two years old, after he had a knock to the teeth and the bleeding wouldn’t stop. “I had to wear one of those foam hats through kindergarten,” he explains.  Like many people with hemophilia, his childhood was punctuated by ‘accidents’. As a teenager, his life was permeated by the forethought needed to manage his condition: > When I got older and into skateboarding, I realized that some of the injuries > I got were quite severe. Every time I did something, I had to plan for the > outcome … and that became daily life for me. > > Martin Nielsen, Denmark, who lives with hemophilia B. Now, as an adult, Martin better understands his internal conflict between the desire to exercise and the threat of bleeds. Despite a love of sports, strenuous activity has come with a price. “It was always two steps forward, one step back, because there was always that little thing that you didn’t recognize [e.g. a bruise] that would turn into a bleeding. Then I would have to rest.”  Trying to build a life on shifting sands  “Studies clearly indicate that seclusion, isolation and depression are more common in people with hemophilia than in those without,” explains Professor Miesbach.  > Naturally, the disease is associated with a strong sense of insecurity and > sometimes even a feeling of withdrawal. Although improvements have been made > in recent years, the quality of life of people with the disease is still > declining compared to the [general] population. > > Wolfgang Miesbach, professor of medicine at Frankfurt University Hospital, > Germany, and member of the executive committee at the European Association for > Haemophilia and Allied Disorders. On his mental health, Martin says: “I think I’ve become a little bit more apathetic … there is a realization that you have a building frame but there isn’t anything to build on. Every time I was trying to build a foundation for that frame, it just crumbled whenever I had an accident or spontaneous bleeding.”  Your voice, your choice: Shared decision-making in hemophilia care  “The term ‘hemophilia-free mind’ was coined in publications, and it means freedom not only from hemophilia itself, but also from the treatment of hemophilia and everything associated with it,” says Professor Miesbach.   For him, consideration of quality of life, mental health and the patient’s holistic needs is essential: > We hardly have a conversation with our patients without mentioning the > improvement in hemophilia therapy and what it means for quality of life, > mental health and this feeling of freedom. > > Wolfgang Miesbach, professor of medicine at Frankfurt University Hospital, > Germany, and member of the executive committee at the European Association for > Haemophilia and Allied Disorders. Martin spoke to his doctor about treatment options and advises others to do the same. He explains that with his hemophilia treatment now, he feels like “I have got my own character back, my own identity back.”   “There is a great need for advancements in hemophilia therapy and a significant demand from patients,” emphasizes Professor Miesbach. “Collaboration with politicians, the pharmaceutical industry, scientific societies, practitioners and patients is essential.”  Recognizing this need, some European countries have already taken the bold step of introducing innovation through individual agreements that benefit both patients and payers. It is incumbent upon all of us, including national authorities, politicians and payers, to make innovative therapies accessible so that patients and clinicians have the full range of treatment choices to meet the diversity of clinical, psychological and personal needs through shared decision-making. The time for action is now to ensure every patient has access to the treatments they need.  -------------------------------------------------------------------------------- 1. Skinner MW. WFH: Closing the global gap – achieving optimal care. Haemophilia. 2012; 18: 1-12. 2. Iorio A, et al. Data and Demographics Committee of the World Federation of Hemophilia. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries. Ann Intern Med. 2019; 171(8): 540-546. 3. Palareti, L., at al. Shared topics on the experience of people with haemophilia living in the UK and the USA and the influence of individual and contextual variables: Results from the HERO qualitative study. Int J Qual Stud Health Well-being. 2015; 10: 28915. 4. Krumb E and Hermans C. Living with a “hemophilia-free mind” – The new ambition of hemophilia care? Res Pract Thromb Haemost. 2021; 5(5): e12567. 5. Mannucci PM. Hemophilia treatment innovation: 50 years of progress and more to come. J Thromb Haemost. 2023; 21 (3): 403-412. 6. Hermans C and Pierce GF. Towards achieving a haemophilia-free mind. Haemophilia. 2023; 29(4): 951-953.

‘PARKINSON’S IS A MAN-MADE DISEASE’ Europe’s flawed oversight of pesticides may be fueling a silent epidemic, warns Dutch neurologist Bas Bloem. His fight for reform pits him against industry, regulators — and time. Text and photos by BARTOSZ BRZEZIŃSKI in Nijmegen, Netherlands Illustration by Laura Scott for POLITICO In the summer of 1982, seven heroin users were admitted to a California hospital paralyzed and mute. They were in their 20s, otherwise healthy — until a synthetic drug they had manufactured in makeshift labs left them frozen inside their own bodies. Doctors quickly discovered the cause: MPTP, a neurotoxic contaminant that had destroyed a small but critical part of the brain, the substantia nigra, which controls movement. The patients had developed symptoms of late-stage Parkinson’s, almost overnight. The cases shocked neurologists. Until then, Parkinson’s was thought to be a disease of aging, its origins slow and mysterious. But here was proof that a single chemical could reproduce the same devastating outcome. And more disturbing still: MPTP turned out to be chemically similar to paraquat, a widely used weedkiller that, for decades, had been sprayed on farms across the United States and Europe. Advertisement While medication helped some regain movement, the damage was permanent — the seven patients never fully recovered. For a young Dutch doctor named Bas Bloem, the story would become formative. In 1989, shortly after finishing medical school, Bloem traveled to the United States to work with William Langston, the neurologist who had uncovered the MPTP-Parkinson’s link. What he saw there reshaped his understanding of the disease — and its causes. “It was like a lightning bolt,” Bloem tells me. “A single chemical had replicated the entire disease. Parkinson’s wasn’t just bad luck. It could be caused.” THE MAKING OF A MAN-MADE DISEASE Today, at 58, Bloem leads a globally recognized clinic and research team from his base at the Radboud University Medical Center in Nijmegen, a medieval Dutch city near the German border. It treats hundreds of patients each year, while the team pioneers studies on early diagnosis and prevention. The hallway outside Bloem’s office was not hectic on my recent visit, but populated — patients moving slowly, deliberately, some with walkers, others with a caregiver’s arm under their own. One is hunched forward in a rigid, deliberate shuffle; another pauses silently by the stairs, his face slack, not absent — just suspended, as if every gesture had become too costly. On its busiest days, the clinic sees over 60 patients. “And more are coming,” Bloem says. Bloem’s presence is both charismatic and kinetic: tall — just over 2 meters, he says with a grin — with a habit of walking while talking, and a white coat lined with color-coded pens. His long, silver-gray hair is swept back, a few strands escaping as he paces the room. Patients paint portraits of him, write poems about him. His team calls him “the physician who never stops moving.” Unlike many researchers of his stature, Bloem doesn’t stay behind the scenes. He speaks at international conferences, consults with policymakers, and states his case to the public as well as to the scientific world. His work spans both care and cause — from promoting movement and personalized treatment to sounding the alarm about what might be triggering the disease in the first place. Alongside his focus on exercise and prevention, he’s become one of the most outspoken voices on the environmental drivers of Parkinson’s — and what he sees as a growing failure to confront their long-term impact on the human brain. Advertisement “Parkinson’s is a man-made disease,” he says. “And the tragedy is that we’re not even trying to prevent it.” When the English surgeon James Parkinson first described the “shaking palsy” in 1817, it was considered a medical curiosity — a rare affliction of aging men. Two centuries later, Parkinson’s disease has more than doubled globally over the past 20 years, and is expected to double again in the next 20. It is now one of the fastest-growing neurological disorders in the world, outpacing stroke and multiple sclerosis. The disease causes the progressive death of dopamine-producing neurons and gradually robs people of movement, speech and, eventually, cognition. There is no cure. Age and genetic predisposition play a role. But Bloem and the wider neurological community contend that those two factors alone cannot explain the steep rise in cases. In a 2024 paper co-authored with U.S. neurologist Ray Dorsey, Bloem wrote that Parkinson’s is “predominantly an environmental disease” — a condition shaped less by genetics and more by prolonged exposure to toxicants like air pollution, industrial solvents and, above all, pesticides. Most of the patients who pass through Bloem’s clinic aren’t farmers themselves, but many live in rural areas where pesticide use is widespread. Over time, he began to notice a pattern: Parkinson’s seemed to crop up more often in regions dominated by intensive agriculture. “Parkinson’s was a very rare disease until the early 20th century,” Bloem says. “Then with the agricultural revolution, chemical revolution, and the explosion of pesticide use, rates started to climb.” Europe, to its credit, has acted on some of the science. Paraquat — the herbicide chemically similar to MPTP — was finally banned in 2007, although only after Sweden took the European Commission to court for ignoring the evidence of its neurotoxicity. Other pesticides with known links to Parkinson’s, such as rotenone and maneb, are no longer approved. Advertisement But that’s not the case elsewhere. Paraquat is still manufactured in the United Kingdom and China, sprayed across farms in the United States, New Zealand and Australia, and exported to parts of Africa and Latin America — regions where Parkinson’s rates are now rising sharply. Once the second-most widely sold herbicide in the world — after glyphosate — paraquat helped drive major profits for its maker, Swiss-based and Chinese-owned company Syngenta. But its commercial peak has long passed, and the chemical now accounts for only a small fraction of the company’s overall business. In the U.S., Syngenta faces thousands of lawsuits from people who say the chemical gave them Parkinson’s. Similar cases are moving ahead in Canada. Syngenta has consistently denied any link between paraquat and Parkinson’s, pointing to regulatory reviews in the U.S., Australia and Japan that found no evidence of causality.  The company told POLITICO that comparisons to MPTP have been repeatedly challenged, citing a 2024 Australian review which concluded that paraquat does not act through the same neurotoxic mechanism. There is strong evidence, the company said in a written response running to more than three pages, that paraquat does not cause neurotoxic effects via the routes most relevant to human exposure — ingestion, skin contact or inhalation. “Paraquat is safe when used as directed,” Syngenta said. Still, for Bloem, even Europe’s bans are no cause for comfort. “The chemicals we banned? Those were the obvious ones,” Bloem says. “What we’re using now might be just as dangerous. We simply haven’t been asking the right questions.” A CHEMICAL EUROPE CAN’T QUIT Among the chemicals still in use, none has drawn more scrutiny — or survived more court battles — than glyphosate. It’s the most widely used herbicide on the planet. You can find traces of it in farmland, forests, rivers, raindrops and even in tree canopies deep inside Europe’s nature reserves. It’s in household dust, animal feed, supermarket produce. In one U.S. study, it showed up in 80 percent of urine samples taken from the general public. For years, glyphosate, sold under the Roundup brand, has been at the center of an international legal and regulatory storm. In the United States, Bayer — which acquired Monsanto, Roundup’s original maker — has paid out more than $10 billion to settle lawsuits linking glyphosate to non-Hodgkin’s lymphoma.  Advertisement Glyphosate is now off-patent and manufactured by numerous companies worldwide. But Bayer remains its top seller — achieving an estimated €2.6 billion in glyphosate-related sales in 2024, even as market competition and legal pressures cut into profits. In Europe, lobbyists for the agricultural and chemical sectors have fought hard to preserve its use, warning that banning glyphosate would devastate farming productivity. National authorities remain split. France has tried to phase it out. Germany has promised a full ban — but never delivered. In 2023 — despite mounting concerns, gaps in safety data and political pressure — the European Union reauthorized it for another 10 years. While most of the debate around glyphosate has centered on cancer, some studies have found possible links to reproductive harm, developmental disorders, endocrine disruption and even childhood cancers. Glyphosate has never been definitively linked to Parkinson’s. Bayer told POLITICO in a written response that no regulatory review has ever concluded any of its products are associated with the disease, and pointed to the U.S.-based Agricultural Health Study, which followed nearly 40,000 pesticide applicators and found no statistically significant association between glyphosate and the disease. Bayer said glyphosate is one of the most extensively studied herbicides in the world, with no regulator identifying it as neurotoxic or carcinogenic. But Bloem argues that the absence of a proven link says more about how we regulate risk than how safe the chemical actually is. Unlike paraquat, which causes immediate oxidative stress and has been associated with Parkinson’s in both lab and epidemiological studies, glyphosate’s potential harms are more indirect — operating through inflammation, microbiome disruption or mitochondrial dysfunction, all mechanisms known to contribute to the death of dopamine-producing neurons. But this makes them harder to detect in traditional toxicology tests, and easier to dismiss. “The problem isn’t that we know nothing,” Bloem says. “It’s that we’re not measuring the kind of damage Parkinson’s causes.” Responding, Bayer pointed to paraquat as one of only two agricultural chemicals that studies have linked directly to the development of Parkinson’s disease — even as Syngenta, its manufacturer, maintains there is no proven connection. Advertisement The EU’s current pesticide evaluation framework, like that of many other regulatory systems, focuses primarily on acute toxicity — short-term signs of poisoning like seizures, sudden organ damage or death. Manufacturers submit safety data, much of it based on animal studies looking for visible behavioral changes. But unlike for the heroin users in California, who were exposed to an unusually potent toxin, Parkinson’s doesn’t announce itself with dramatic symptoms in the short term. It creeps in as neurons die off, often over decades. “We wait for a mouse to walk funny,” Bloem says. “But in Parkinson’s, the damage is already done by the time symptoms appear.” The regulatory tests also isolate individual chemicals, rarely examining how they interact in the real world. But a 2020 study in Japan showed how dangerous that assumption may be. When rodents were exposed to glyphosate and MPTP — the very compound that mimicked Parkinson’s in the California heroin cases — the combination caused dramatically more brain cell loss than either substance alone. “That’s the nightmare scenario,” Bloem says. “And we’re not testing for it.” Even when data does exist, it doesn’t always reach regulators. Internal company documents released in court suggest Syngenta knew for decades that paraquat could harm the brain — a charge the company denies, insisting there is no proven link. More recently, Bayer and Syngenta have faced criticism for failing to share brain toxicity studies with EU authorities in the past — data they had disclosed to U.S. regulators. In one case, Syngenta failed to disclose studies on the pesticide abamectin. The Commission and the EU’s food and chemical agencies have called this a clear breach. Bloem sees a deeper issue. “Why should we assume these companies are the best stewards of public health?” he asked. “They’re making billions off these chemicals.”  Syngenta said that none of the withheld studies related to Parkinson’s disease and that it has since submitted all required studies under EU transparency rules. The company added that it is “fully aligned with the new requirements for disclosure of safety data.” Some governments are already responding to the links between Parkinson’s and farming. France, Italy and Germany now officially recognize Parkinson’s as a possible occupational disease linked to pesticide exposure — a step that entitles some affected farmworkers to compensation. But even that recognition, Bloem argues, hasn’t forced the broader system to catch up. WHERE SCIENCE STOPS, POLITICS BEGINS Bloem’s mistrust leads straight to the institutions meant to protect public health — and to people like Bernhard Url, the man who has spent the past decade running one of the most important among them.  Url is the outgoing executive director of the European Food Safety Authority, or EFSA — the EU’s scientific watchdog on food and chemical risks, based in Parma, Italy. The agency has come under scrutiny in the past over its reliance on company-submitted studies. Url doesn’t deny that structure, but says the process is now more transparent and scientifically rigorous. I met Url while he was on a visit to Brussels, during his final months as EFSA’s executive director. Austrian by nationality and a veterinarian by training, he speaks precisely, choosing his words with care. If Bloem is kinetic and outwardly urgent, Url is more reserved — a scientist still operating within the machinery Bloem wants to reform. Advertisement Still, Url didn’t dispute the core of the critique. “There are areas we don’t yet take into consideration,” he told me, pointing to emerging science around microbiome disruption, chemical synergy and chronic low-dose exposure. He didn’t name Parkinson’s, but the implications were clear. “We’re playing catch-up,” he admitted. Part of the problem, he suggested, is structural. The agency relies on a system built around predefined methods and industry-supplied data. “We assess risk based on what we’re given, and what the framework allows us to assess,” Url said. “But science evolves faster than legislation. That’s always the tension.” EFSA also works under constraints that its pharmaceutical counterpart, the European Medicines Agency, does not. “EMA distributes money to national agencies,” Url said. “We don’t. There’s less integration, less shared work. We rely on member states volunteering experts. We’re not in the same league.” A pesticide-free farm in in Gavorrano, Italy. | Alberto Pizzoli/AFP via Getty Images Url didn’t sound defensive. If anything, he sounded like someone who’s been pushing against institutional gravity for a long time. He described EFSA as an agency charged with assessing a food system worth trillions — but working with limited scientific resources, and within a regulatory model that was never designed to capture the risks of chronic diseases like Parkinson’s. “We don’t get the support we need to coordinate across Europe,” he said. “Compared to the economic importance of the whole agri-food industry … it’s breadcrumbs.” But he drew a sharp line when it came to responsibility. “The question of what’s safe enough — that’s not ours to answer,” he said. “That’s a political decision.” EFSA can flag a risk. It’s up to governments to decide whether that risk is acceptable. Advertisement It was a careful way of saying what Bloem had said more bluntly: Science may illuminate the path, but policy chooses where — and whether — to walk it. And in a food system shaped by powerful interests, that choice is rarely made in a vacuum. “There are gaps,” Url said, “and we’ve said that.” But gaps in science don’t always lead to action. Especially when the cost of precaution is seen as an economic threat. THE DOCTOR WHO WON’T SLOW DOWN Evidence from the field is becoming harder to ignore. In France, a nationwide study found that Parkinson’s rates were significantly higher in vineyard regions that rely heavily on fungicides. Another study found that areas with higher agricultural pesticide use — often measured by regional spending — tend to have higher rates of Parkinson’s, suggesting a dose-response relationship. In Canada and the U.S., maps of Parkinson’s clusters track closely with areas of intensive agriculture. The Netherlands has yet to produce comparable data. But Bloem believes it’s only a matter of time. “If we mapped Parkinson’s here, we’d find the same patterns,” he says. “We just haven’t looked yet.” In fact, early signs are already emerging. The Netherlands, known for having one of the highest pesticide use rates in Europe, has seen a 30 percent rise in Parkinson’s cases over the past decade — a slower increase than in some other regions of the world, but still notable, Bloem says. In farming regions like the Betuwe, on the lower reaches of the Rhine River, physiotherapists have reported striking local clusters. One village near Arnhem counted over a dozen cases. “I don’t know of a single farmer who’s doing things purposely wrong,” Bloem says. “They’re just following the rules. The problem is, the rules are wrong.” To Bloem, reversing the epidemic means shifting the regulatory mindset from reaction to prevention. That means requiring long-term neurotoxicity studies, testing chemical combinations, accounting for real-world exposure, genetic predisposition and the kind of brain damage Parkinson’s causes — and critically, making manufacturers prove safety, rather than scientists having to prove harm. “We don’t ban parachutes after they fail,” Bloem says. “But that’s what we do with chemicals. We wait until people are sick.” Advertisement His team is also studying prevention-focused interventions — including exercise, diet and stress reduction — in people already diagnosed with Parkinson’s, in one of the most comprehensive trials of its kind. Still, Bloem is realistic about the limits of individual action. “You can’t exercise your way out of pesticide exposure,” he says. “We need upstream change.” Bloem has seen it before — the same pattern playing out in slow motion. “Asbestos,” he says “Lead in gasoline. Tobacco. Every time, we acted decades after the damage was done.” The science existed. The evidence had accumulated. But the decision to intervene always lagged. “It’s not that we don’t know enough,” he adds. “It’s that the system is not built to listen when the answers are inconvenient.” The clinic has grown quiet. Most of the staff have left for the day, the corridors are still. Bloem gathers his things, but he’s not finished yet. One more phone call to make — something he’ll take, as always, while walking. As we stand up to go into the hallway, he pauses. “If we don’t fix this now,” he says, “we’re going to look back in 50 years and ask: ‘What the hell were we thinking?’” He slips on a pair of black headphones, nods goodbye and turns toward the exit. Outside, he’s already striding across the Radboud campus, talking into the cold evening air — still moving, still making calls, still trying to bend a stubborn system toward change. Graphics by Lucia Mackenzie.

Sometimes statistics don’t tell the full story. This is particularly true if you look at EFPIA’s recently published European Economic Impact report, which looks at the contribution of the pharmaceutical industry to Europe’s economy, as well as those of other regions. The report finds “a strong and growing sector”. It shows that research and development (R&D) expenditure in Europe has grown on average by 4.4 percent each year between 2010 and 2022, from €27.8 billion to €46.2 billion. Good news? Yes and no. If we look at the wider picture, Europe’s position is clearly becoming more and more precarious against a backdrop of falling competitiveness. While global research into medicines and vaccines is growing, Europe’s share of the pie is getting smaller as the distribution of R&D shifts elsewhere. > While global research into medicines and vaccines is growing, Europe’s share > of the pie is getting smaller as the distribution of R&D shifts elsewhere. R&D spending in Europe is consistently outpaced by the US and increasing competition from China. In the US, R&D investment grew by 5.4 percent between 2010 and 2022 (from €30.7 billion to €71.5 billion) and five times greater (20.7 percent) in China (from €1.4 billion to €14.8 billion). If we look at Europe first: In 2022, global R&D spending in the sector was €143.6 billion; Europe accounted for 32.2 percent of this. As a sector, we contribute more to the EU trade balance than any other. Added to this, 16 of the world’s top 50 life sciences universities are based here. * In 2022, the industry contributed €311 billion to the EU — 2.0 percentof its gross value-added (GVA). * The industry supports 2.3 million jobs in the EU — a 2.1 percent increase year on year between 2016 to 2022. * Driven by consistently high levels of research and innovation — and a GVA of €197,000 per worker — the pharmaceutical industry in Europe is three times as productive as the European economy as a whole. However, the report also found that in the US, for example, GVA per hour per worker is double that of the EU. China’s growth is one of a number of trends that is now shown to correlate with a relative decline in the number of new molecule entities (NME) discovered in Europe. The EU fell behind China for NME discovered in 2023. NMEs are drugs with an active ingredient, marketed for the first time, and vital to R&D activity. The report suggests that China’s life sciences boom is due to its dedication to improving its regulatory environment, increased funding streams and strategic investment in advanced technologies. This is clearly paying off, and while it might send shockwaves through Europe, lessons can be learned. As other regions ramp up their investments and streamline regulatory frameworks, Europe cannot afford to become a secondary or even tertiary player in global health innovation. > As other regions ramp up their investments and streamline regulatory > frameworks, Europe cannot afford to become a secondary or even tertiary player > in global health innovation. Europe’s fragmented regulatory environment exacerbates the gap, reducing the EU’s competitiveness as a location for clinical trials. Recent research showed that 60,000 fewer patients living in Europe had access to clinical trials since 2018, missing out on new research in immunisation, cancer, rare diseases and paediatrics. Europe’s global share of commercial trials — those sponsored and funded by a pharmaceutical company — is half of what it was a decade ago. Meanwhile, commercial trials in China rocketed, making up 18 percent of those taking place globally. For the most innovative therapeutics, advanced cell and gene therapies, China holds a 42 percent share, the biggest in the world. The impact of Europe’s decline reverberates beyond patient care. Skills and expertise are migrating — nearly three-quarters of European science graduates choose to remain in the US after completing their PhDs. Over time we stand to lose our ability to recruit and retain the next generation of scientists. > In Europe we have fantastic ideas, the skills, world-class academic > institutions and pockets of brilliance, but our processes are slow, and our > eco-system is very fragmented. In Europe we have fantastic ideas, the skills, world-class academic institutions and pockets of brilliance, but our processes are slow, and our eco-system is very fragmented. The scale and pace at which we are losing global share of research shows there is only a finite amount of time to turn things around. What needs to happen? Pharmaceutical companies support local economies, boost regional development, create highly skilled jobs, and bring funding to hospitals and research centres. In fact, the data shows that productivity per worker in the pharmaceutical sector is higher than in the economy as a whole. The recognition of the importance of the sector by EU leaders is a positive step. However, overcoming Europe’s widening competitiveness gap needs immediate and robust action now. Mario Draghi’s much anticipated report on how to boost Europe’s competitiveness makes for hopeful reading. The revision of the Pharmaceutical Legislation offers the opportunity to update a 20-year-old regulatory framework to create a unified and streamlined regulatory environment to foster innovation and competitiveness in Europe, on a par with the Food and Drug Administration, among other regulatory bodies. The industry has also outlined a detailed roadmap for policymakers through a Strategy for European Life Sciences, aligning closely with recommendations from the Draghi and Letta reports, which emphasize the urgent need to power up Europe’s innovation capacity. Together with insights from the EFPIA’s Clinical Trials Ecosystem Report, these recommendations form a comprehensive approach to addressing regulatory, investment and operational challenges, setting the foundation for Europe to secure its position as a global leader in health innovation. Europe has the potential. And now the ambition. It is time to turn things around.

The European Centre for Disease Prevention and Control is considering quitting Elon Musk’s social media platform X, its director said Tuesday. Speaking at POLITICO’s Health Care Summit in Brussels, ECDC Director Pamela Rendi-Wagner said social media is a “breeding ground for mis- and disinformation.” “Twitter, or X, is now in every newspaper. Everybody is leaving X, and maybe for good reasons. We are discussing this as well at ECDC at the moment,” she said. “Everybody does, why shouldn’t we,” she added. Speaking about scientific misinformation on social media, Rendi-Wagner said that trust in institutions was lost during the Covid-19 pandemic. “When people don’t understand scientific evidence-based messages, they get scared and turn to other sources of information,” she said, adding that for these reasons the agency is working on avoiding scientific language in their reports. “We have entered a new time,” she said, and added that scientific institutions must find new ways of advising people what to do for their well-being. “We cannot just have the warning index finger and say ‘you must do this,’ because people don’t want that,” she said. “I think we should find new ways of motivating them in a positive way to take the right decisions for themselves, their families and their communities,” she added. However, “I think we cannot only say social media is bad, because we should take advantage of social media [to] engage also with the communities and the people,” she said.

References [1] Fabry Disease. Cleveland Clinic. https://my.clevelandclinic.org/health/diseases/16235-fabry-disease  [2] Familial Hemophagocytic Lymphohistiocytosis. National Library of Medicine. https://www.ncbi.nlm.nih.gov/books/NBK1444/  [3] Autosomal recessive congenital ichthyosis 4B. National Library of Medicine.  https://www.ncbi.nlm.nih.gov/medgen/108615 [4] European Reference Networks. European Commission. https://health.ec.europa.eu/rare-diseases-and-european-reference-networks/european-reference-networks_en  [5] CRA Insights. A Landscape Assessment of Newborn Screening (NBS) in Europe. Charles River Associates. https://media.crai.com/wp-content/uploads/2021/11/28135510/CRA-Insights-NBS-Policy-Updated-28-February-2024-vSTCCR.pdf 

Without doubt, when we reflect on the many achievements of the UK healthcare system and our NHS, there is much to be proud of – but today we are faced with a paradox. We are living in a time where breakthrough science has the potential to transform treatment options for some of the toughest health conditions faced by patients in the UK. Yet the question is whether the system is currently set up to match the pace of innovation that science provides, so patients can fully benefit? At Johnson & Johnson, our teams are continuously working to get ahead of the most complex diseases affecting patients and their families, but we know that these treatments only matter if patients can access them when they need them. > The question is whether the system is currently set up to match the pace of > innovation that science provides, so patients can fully benefit? The last decade has seen a series of changes to the UK access environment for new medicines that have made it increasingly challenging for the NHS to deliver the innovative care that patients need. Right now, just 56% of all new medicines approved by the European Medicines Agency (EMA) are available to patients in England and only 54% in Scotland. This compares with 88% in Germany and 77% in Italy.[i]     Without bold reform, access to medicines will continue to stagnate in the UK, risking our position as a leading destination to do life sciences. We are already witnessing the impact. The government’s Office for Life Sciences charts that foreign direct investment in the UK life sciences sector more than halved between 2021 and 2023.[ii] Now with a new government in place, we have an unparalleled opportunity to strengthen collaboration across the life sciences community, achieve our shared ambitions for the sector and truly deliver the best possible care for every patient in the UK. The good news is that we have a strong recent precedent to draw upon. The UK’s COVID-19 vaccination programme showed what’s possible when political leaders, the pharmaceutical industry and health systems work together. By doing so, we were able to bring innovation directly into the hands of healthcare providers, deploy the nation’s resources more effectively and, most importantly of all, transform outcomes for patients. The challenge now is to build on the lessons learned during the pandemic. Even the most pioneering drugs and therapies are only valuable if patients can actually be treated with them. Going forward, it is a certainty that UK policymakers must prioritise a shared vision and joint action to ensure the NHS can deliver “the best that modern science can offer”. [iii] > Even the most pioneering drugs and therapies are only valuable if patients can > actually be treated with them. Bodies that assess new medicines for adoption by health systems, such as The National Institute for Health and Care Excellence (NICE) for NHS England, must strike a tricky balance. Appraisal frameworks need to be robust and inclusive while keeping pace with exciting scientific developments and evolving treatment pathways. However, in my opinion, a recent review of NICE’s methods[iv] was a missed opportunity to incorporate more insight from the life sciences sector and introduce greater flexibility into the system. For one, severity modifiers were introduced. These enable adjustments to the thresholds at which medicines for particularly debilitating conditions are assessed. While these modifiers could benefit a wider range of patients, their rigid, formulaic criteria may unintentionally limit access to treatments for those with the most severe conditions. To highlight another example, an increasing number of innovative medicines are effective across multiple rare diseases or cancers.[v] Unfortunately, the current criteria NHS England relies upon does not straightforwardly evaluate the differential value that such multi-indication medicines provide. If a clear and accessible route were established to include indication-based pricing, it could make it easier for these critical new medicines to be recommended for use and for more patients to receive their full benefit. There’s real urgency when we’re talking about access to new medicines in the UK. Currently, just 25% of new oncology medicines approved by the EMA between 2019 and 2022 are fully available on the NHS in England.i This means that the most effective treatment may simply be unavailable for some patients, not due to its efficacy, but because of where they live. Timely access to the right treatment does two things, it keeps people healthy and prevents disease worsening so they can participate in society and a thriving economy. It also impacts patient outcomes and reduces the likelihood of co-morbidity. As highlighted in the September 2024 Lord Darzi report, by improving access to care and addressing long-term sickness the NHS plays a role in driving national prosperity.[vi] The recently announced Voluntary Scheme for Branded Medicine Pricing, Access and Growth (VPAG) Investment Programme is certainly a step in the right direction. Of the £400 million investment secured, 5% will focus on modernising the access environment.[vii] It will be critical for this investment to deliver meaningful change that ensures the UK avoids repeatedly falling behind when it comes to accessing the medicines of the future. Over and above individual policy and regulatory changes, the path to lasting improvements in the access landscape lies with all parts of the life sciences ecosystem working together to fuel a virtuous cycle of innovation. Only by actively working together – government, healthcare providers and industry – can we create an environment that fosters innovation and, more importantly, brings its benefits to patients. This year, ‎‎ ‎Johnson & Johnson proudly celebrated 100 years of operations in the UK. Our expertise has served as the foundation for decades of successful partnership with patients, healthcare providers, clinical researchers and the NHS. That’s why we wholeheartedly welcome the new government’s ambition to collaborate with the private sector on life sciences innovation. After all, healthy people build healthy societies and healthy economies. > …The path to lasting improvements in the access landscape lies with all parts > of the life sciences ecosystem working together to fuel a virtuous cycle of > innovation. Follow Johnson & Johnson Innovative Medicine UK on LinkedIn for updates on our business, our people and our community -------------------------------------------------------------------------------- [i] EFPIA (2024). EPFIA Patients W.A.I.T. Indicator 2023 Survey. Available here: https://efpia.eu/media/vtapbere/efpia-patient-wait-indicator-2024.pdf. Accessed September 2024. [ii] HM Government (2024). Life Sciences Competitiveness Indicators 2024: summary. Available here: https://www.gov.uk/government/publications/life-sciences-sector-data-2024/life-sciences-competitiveness-indicators-2024-summary. Accessed September 2024. [iii] The Labour Party (2024). Labour’s Manifesto: Build an NHS fit for the future. Available here: https://labour.org.uk/change/build-an-nhs-fit-for-the-future/. Accessed September 2024. [iv] NICE (2024). Public board meetings – NICE methods agenda board paper. Available here: https://www.nice.org.uk/get-involved/meetings-in-public/public-board-meetings/agenda-and-papers-march-2024. Accessed September 2024. [v] Mestre-Ferrandiz, J., Towse, A., Dellamano, R. and Pistollato, M. (2015) Multi-indication Pricing: Pros, Cons and Applicability to the UK. OHE Seminar Briefing. Available here: https://www.ohe.org/publications/multi-indication-pricing-pros-cons-and-applicability-uk/. Accessed September 2024. [vi] Lord Ari Darzi (2024). Independent Investigation of the National Health Service in England. Available here: https://assets.publishing.service.gov.uk/media/66e1b49e3b0c9e88544a0049/Lord-Darzi-Independent-Investigation-of-the-National-Health-Service-in-England.pdf. Accessed September 2024. [vii] HM Government (2024). UK secures £400 million investment to boost clinical trials [Press release]. Available here: https://www.gov.uk/government/news/uk-secures-400-million-investment-to-boost-clinical-trials. Accessed September 2024. CP-476396 | September 2024